Italfarmaco Touts Positive Results in Phase III DMD Trial Ahead of PDUFA

DMD Child in wheelchair_iStock, Prostock-Studio

Pictured: Boy in a wheelchair looking out a window/iStock, Prostock-Studio

Italian pharma company Italfarmaco on Tuesday reported the full Phase III results for its drug candidate givinostat in patients with Duchenne muscular dystrophy, which reached the primary endpoint.

According to the results posted in The Lancet Neurology, Italfarmaco’s Epidys trial met the primary endpoint of a change in four-stair climb assessment from the baseline to 72 weeks. Reaching this point showed the givinostat can delay disease progression when added to corticosteroid treatment.

“The results of the Phase III Epidys study are encouraging and highlight the dedication of Italfarmaco’s research and clinical teams to achieve this milestone for the company,” Italfarmaco Group CMO Paolo Bettica said in a statement. “We want to thank all the individuals and their families who participated in this international study and would also like to acknowledge the tireless efforts of the clinical investigators and the unwavering support of the DMD community.”

The trial enrolled 179 ambulant male patients six years of age and older with DMD across North America and Europe. Italfarmaco said that the patients who received the drug showed a slower decline in performing the functional task than those who received the placebo. The difference versus the placebo group was 1.78 seconds and earned a p-value of 0.037.  

For the secondary endpoints, the company said that some of the ones that investigated muscle function and strength had shown favorable results in the givinostat cohort. The drug was associated with a 40% less decline in the NSAA total score and item loss, showing a delay in disease progression. The vastus lateralis fat fraction (VLFF), a predictor for the loss of ambulation and disease progression, showed a 30% reduction against the placebo. 

The most common related adverse event was decreased platelet count, hypertriglyceridemia, abdominal pain and diarrhea. No withdrawals due to treatment were recorded. 

The results are critical for Italfarmaco as the FDA intends to hand down its verdict on givinostat on March 21. The drug is designed to be given orally and is intended to block the enzymes histone deacetylases, or HDACs, which mainly affect muscle gene expression.

The pharma originally submitted its NDA for givinostat in June 2023, with the initial topline data disclosed in 2022.

In the DMD market, Sarepta’s treatment Elevidys has been a significant earner, bringing in over $200 million since its launch in the middle of last year.

Tyler Patchen is a staff writer at BioSpace. You can reach him at Follow him on LinkedIn.

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